A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Assess the Efficacy and Safety of CEP-1347 in Patients With Parkinson's Disease
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 806
- 试验地点
- 66
- 主要终点
- Number of participants with disability using United Parkinson's Disease Rating Scale (UPDRS)
研究概览
简要总结
The purpose of this study is to establish safety for CEP-1347 and to determine an efficacious dose in the treatment of Parkinson's disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 30 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients will be included in the study if all of the following criteria are met:
- •Willing and able to give informed consent
- •Age 30 years or older at time of diagnosis of Parkinson's disease
- •Have idiopathic Parkinson's disease with at least 2 cardinal signs of disease: resting tremor, bradykinesia, or rigidity
- •Modified Hoehn and Yahr stage less than or equal to 2.5
- •Must have had screening procedures for cancer appropriate for the patient's age and gender, within the last 12 months; or be willing to obtain such screening before randomization
- •Women: are not breastfeeding
- •Women: nonchildbearing potential (ie, postmenopausal or surgically sterile) or must use a medically accepted contraceptive regimen for at least 60 days before the baseline visit, and agree to continue such use throughout the duration of the study and for 30 days after the final dose of study drug. Women must be given a pregnancy test unless they are at least 2 years postmenopausal or surgically sterile.
排除标准
- •Patients will be excluded from participating in this study if 1 or more of the following criteria are met:
- •Have atypical Parkinsonism due to drugs, metabolic disorders, encephalitis, or other neurodegenerative diseases
- •Have confirmed diagnosis of Parkinson's disease for more than 5 years
- •Have a tremor score of 3 or more in any body part
- •Have any other known medical or psychiatric condition that may compromise participation in the study
- •Have a history of prior malignancy (excluding basal or squamous cell cancer of the skin) within the previous 5 years
- •Have an unresolved abnormal cancer screening test result before randomization
- •Have greater than trace amounts of glycosuria at screening, except for known diabetic patients
- •Have estimated creatinine clearance less than 50 mL/min
- •Have liver function tests (LFT) greater than 3 times the upper limit of normal (ULN)
- •Have any other clinically significant ECG or laboratory finding
- •Have any history of malignant melanoma
- •Have history of seizures (except febrile) or posttraumatic epilepsy
- •Have Mini-Mental State Exam (MMSE) score ≤ 26
- •Have taken another investigational drug within 60 days before the baseline visit
- •Have received prior treatment with CEP-1347
- •Have received treatment with agents with potentially confounding anti-Parkinson's disease effects, with specified substrates for CYP3A4/5, or with inhibitors of CYP3A4/5
- •Received treatment within 6 months before the baseline visit with agents that may induce Parkinson's disease
- •Are expected, within the next 3 months, to reach a level of disability sufficient to require dopaminergic therapy
- •Have BECK depression score ≥ 15
- •Have known or suspected sensitivity to the investigational study drugs, including B-CIT
研究组 & 干预措施
CEP-1347 10mg
CEP-1347 was administered at a dosage of 10mg twice daily (bid); capsule strengths were 5, 12.5, and 25 mg. Each patient took 2 capsules at each dosing time, approximately 12 hours apart, within 30 minutes after the morning and evening meals) for a total of 4 capsules per day.Patients were randomly assigned to CEP-1347 or placebo treatment in a 1:1:1:1 ratio. A blocked randomization scheme was used to ensure approximately equal numbers of patients in each of the 4 treatment groups at each center.
干预措施: CEP-1347 10mg (Drug)
CEP-1347 25mg
CEP-1347 was administered at a dosage of 25mg twice daily (bid); capsule strengths were 5, 12.5, and 25 mg. Each patient took 2 capsules at each dosing time, approximately 12 hours apart, within 30 minutes after the morning and evening meals) for a total of 4 capsules per day.Patients were randomly assigned to CEP-1347 or placebo treatment in a 1:1:1:1 ratio. A blocked randomization scheme was used to ensure approximately equal numbers of patients in each of the 4 treatment groups at each center.
干预措施: CEP1347 25mg (Drug)
CEP-1347 50mg
CEP-1347 was administered at a dosage of 50mg twice daily (bid); capsule strengths were 5, 12.5, and 25 mg. Each patient took 2 capsules at each dosing time, approximately 12 hours apart, within 30 minutes after the morning and evening meals) for a total of 4 capsules per day.Patients were randomly assigned to CEP-1347 or placebo treatment in a 1:1:1:1 ratio. A blocked randomization scheme was used to ensure approximately equal numbers of patients in each of the 4 treatment groups at each center.
干预措施: CEP-1347 50mg (Drug)
Placebo
Placebo capsules matching the CEP-1347 capsules were administered in the same manner.
干预措施: Placebo Comparator (Other)
结局指标
主要结局
Number of participants with disability using United Parkinson's Disease Rating Scale (UPDRS)
时间窗: 48 months
Number of participants with disability sufficient to require dopaminergic therapy was assessed according to the United Parkinson's Disease Rating Scale (UPDRS) Parts I and II are historical data and are designed to rate mentation, behavior and mood; Part III is done as a motor examination at the time of a visit. The UPDRS measures patient status on a scale 0, which is normal or none, to 4, which is severe or the worst scenario.
次要结局
- Change from Baseline to 22 months in ([123I]β-CIT) Uptake Participants(Change from Baseline to 22 months)
- Safety and Tolerability as assessed by the number of participants experiencing adverse events(48 months)